2025
Targeted inhibition of pathobiont virulence factor mitigates alcohol-associated liver disease
Yang Y, Duan Y, Lang S, Fondevila M, Schöler D, Harberts A, Cabré N, Chen S, Shao Y, Vervier K, Miyamoto Y, Zhang X, Chu H, Yang L, Tan C, Eckmann L, Bosques-Padilla F, Verna E, Abraldes J, Brown R, Vargas V, Altamirano J, Caballería J, Shawcross D, Louvet A, Lucey M, Mathurin P, Garcia-Tsao G, Bataller R, Stärkel P, Lawley T, Schnabl B. Targeted inhibition of pathobiont virulence factor mitigates alcohol-associated liver disease. Cell Host & Microbe 2025, 33: 957-972.e6. PMID: 40441146, PMCID: PMC12162233, DOI: 10.1016/j.chom.2025.05.003.Peer-Reviewed Original ResearchConceptsEthanol-induced liver diseaseAlcohol-associated liver diseaseAlcohol-associated hepatitisLiver diseaseGenome of Escherichia coliE. coliMetagenomic sequencing of fecal samplesInternational cohort of patientsGenetic manipulation of bacteriaGnotobiotic mouse modelOutcomes of patientsManipulation of bacteriaCohort of patientsScavenger receptor MARCOGlobal health burdenVirulence factorsMetagenomic sequencingGut microbiotaGenetic manipulationDisease progressionMouse modelKupffer cellsKpsMBacterial spreadInternational cohortCharacterizing the ADPKD-IFT140 Phenotypic Signature with Deep Learning and Advanced Imaging Biomarkers
Ghanem A, Debeh F, Borghol A, Zagorec N, Tapia A, Smith B, Paul S, Basit A, AlKhatib B, Nader N, Antoun M, Gregory A, Yang H, Schauer R, Dahl N, Hanna C, Torres V, Kline T, Harris P, Gall E, Chebib F. Characterizing the ADPKD-IFT140 Phenotypic Signature with Deep Learning and Advanced Imaging Biomarkers. Kidney International Reports 2025 DOI: 10.1016/j.ekir.2025.04.062.Peer-Reviewed Original ResearchArea under the curveHeight-adjusted total kidney volumeAutosomal dominant polycystic kidney diseaseEstimated glomerular filtrationDisease-causing variantsDevelopment cohortAdvanced imaging biomarkersTotal kidney volumeDominant polycystic kidney diseaseRetrospective cohort studyPolycystic kidney diseaseClinical presentationCyst volumeKidney volumeCyst diameterCystic indexDiverse clinical settingsLiver cystsCohort studyExternal cohortKidney diseaseLarge cystsInternational cohortGlomerular filtrationPatientsIMPROVED OUTCOMES IN BCG-UNRESPONSIVE BLADDER CANCER PATIENTS WITH NON-INVASIVE DISEASE UNDERGOING BLADDER SPARING THERAPY COMPARED TO T1 DISEASE: RESULTS FROM AN INTERNATIONAL COHORT
Annapureddy D, Taylor J, Howard J, Tan W, McElree I, Davaro F, Yim K, Harrington S, Dyer E, Black A, Kanabur P, Roumiguié M, Lerner S, Black P, Raman J, Preston M, Steinberg G, Huang W, Li R, Packiam V, O'Donnell M, Kamat A, Woldu S, Lotan Y. IMPROVED OUTCOMES IN BCG-UNRESPONSIVE BLADDER CANCER PATIENTS WITH NON-INVASIVE DISEASE UNDERGOING BLADDER SPARING THERAPY COMPARED TO T1 DISEASE: RESULTS FROM AN INTERNATIONAL COHORT. Urologic Oncology Seminars And Original Investigations 2025, 43: 80-81. DOI: 10.1016/j.urolonc.2024.12.203.Peer-Reviewed Original ResearchBCG-unresponsive NMIBCNon-muscle invasive bladder cancerBladder sparing therapyAll-Cause MortalityOncological outcomesRadical cystectomyCancer-specific mortalityNon-invasive diseaseBCG-unresponsiveBladder cancerPathological outcomesT1 diseaseT1 patientsCIS patientsMedian timeProgression to muscle-invasive bladder cancerBCG-unresponsive NMIBC patientsImpact of pathological stageImpact of tumor stageInternational cohortMuscle-invasive bladder cancerAssociated with significant morbiditySingle-institution seriesHeterogeneous group of patientsCompare oncologic outcomes
2024
Docetaxel Versus Androgen-Receptor Signaling Inhibitors (ARSI) as Second-Line Therapy After Failure of First-Line Alternative ARSI for the Elderly ≥ 75 Years Old With Metastatic Castration-Resistant Prostate Cancer (mCRPC): A SPARTACUSS—Meet-URO 26 Real-World Study
Patrikidou A, Saieva C, Lee-Ying R, Nuzzo P, Zarif T, McClure H, Davidsohn M, Eid M, Spinelli G, Catalano F, Cremante M, Fotia G, Rossetti S, Valenca L, Vauchier C, Ottanelli C, Andrade L, Gennusa V, Mestre R, Fornarini G, Pignata S, Procopio G, Santini D, Ravi P, Sweeney C, Heng D, De Giorgi U, Fizazi K, Russo A, Francini E, Group S. Docetaxel Versus Androgen-Receptor Signaling Inhibitors (ARSI) as Second-Line Therapy After Failure of First-Line Alternative ARSI for the Elderly ≥ 75 Years Old With Metastatic Castration-Resistant Prostate Cancer (mCRPC): A SPARTACUSS—Meet-URO 26 Real-World Study. Clinical Genitourinary Cancer 2024, 22: 102230. PMID: 39461026, DOI: 10.1016/j.clgc.2024.102230.Peer-Reviewed Original ResearchConceptsMetastatic castration-resistant prostate cancerAndrogen receptor signaling inhibitorsCastration-resistant prostate cancerAbiraterone acetateOverall survivalProstate cancerElderly patientsSignaling InhibitorsCohort of consecutive patientsSecond-line therapyStandard first-lineReal-world studySecond-lineConsecutive patientsTreatment toxicityFirst-lineD. CONCLUSIONSPatient comorbiditiesIdentified patientsRetrospective designToxicity outcomesD groupInternational cohortPatientsNo differenceNeurostructural subgroup in 4291 individuals with schizophrenia identified using the subtype and stage inference algorithm
Jiang Y, Luo C, Wang J, Palaniyappan L, Chang X, Xiang S, Zhang J, Duan M, Huang H, Gaser C, Nemoto K, Miura K, Hashimoto R, Westlye L, Richard G, Fernandez-Cabello S, Parker N, Andreassen O, Kircher T, Nenadić I, Stein F, Thomas-Odenthal F, Teutenberg L, Usemann P, Dannlowski U, Hahn T, Grotegerd D, Meinert S, Lencer R, Tang Y, Zhang T, Li C, Yue W, Zhang Y, Yu X, Zhou E, Lin C, Tsai S, Rodrigue A, Glahn D, Pearlson G, Blangero J, Karuk A, Pomarol-Clotet E, Salvador R, Fuentes-Claramonte P, Garcia-León M, Spalletta G, Piras F, Vecchio D, Banaj N, Cheng J, Liu Z, Yang J, Gonul A, Uslu O, Burhanoglu B, Uyar Demir A, Rootes-Murdy K, Calhoun V, Sim K, Green M, Quidé Y, Chung Y, Kim W, Sponheim S, Demro C, Ramsay I, Iasevoli F, de Bartolomeis A, Barone A, Ciccarelli M, Brunetti A, Cocozza S, Pontillo G, Tranfa M, Park M, Kirschner M, Georgiadis F, Kaiser S, Van Rheenen T, Rossell S, Hughes M, Woods W, Carruthers S, Sumner P, Ringin E, Spaniel F, Skoch A, Tomecek D, Homan P, Homan S, Omlor W, Cecere G, Nguyen D, Preda A, Thomopoulos S, Jahanshad N, Cui L, Yao D, Thompson P, Turner J, van Erp T, Cheng W, Feng J. Neurostructural subgroup in 4291 individuals with schizophrenia identified using the subtype and stage inference algorithm. Nature Communications 2024, 15: 5996. PMID: 39013848, PMCID: PMC11252381, DOI: 10.1038/s41467-024-50267-3.Peer-Reviewed Original ResearchConceptsGray matter changesDisorder constructsEnlarged striatumPsychiatric conditionsMental disordersSubcortical regionsSchizophreniaBiological foundationsMatter changesBrain imagingStriatumDisordersBiological factorsIndividualsSubtypesHealthy subjectsCross-sectional brain imagingHippocampusTemporal trajectoriesInternational cohortSubgroup 2Subgroup 1SubgroupsDesigning medical artificial intelligence systems for global use: focus on interoperability, scalability, and accessibility
Oikonomou E, Khera R. Designing medical artificial intelligence systems for global use: focus on interoperability, scalability, and accessibility. Hellenic Journal Of Cardiology 2024, 81: 9-17. PMID: 39025234, DOI: 10.1016/j.hjc.2024.07.003.Peer-Reviewed Original ResearchArtificial intelligenceMedical artificial intelligence systemsDesigning AI systemsMachine learning systemsArtificial intelligence systemsBenefits of AIIntelligent systemsAI systemsLearning systemEnd-usersData typesAI developmentInteroperabilityTemporal settingAccessScalabilityTreatment of cardiovascular diseasesIntelligenceSystemMachineQuality assuranceInternational cohortCardiovascular diseaseObstaclesExternal validation of the SI2NCAL2C score for outcomes following cerebral venous thrombosis
Klein P, Shu L, Lindgren E, de Winter M, Siegler J, Simpkins A, Omran S, Heldner M, de Havenon A, Abdalkader M, Al Kasab S, Stretz C, Wu T, Wilson D, Asad S, Almallouhi E, Frontera J, Kuohn L, Rothstein A, Bakradze E, Henninger N, Zubair A, Sharma R, Kerrigan D, Aziz Y, Mistry E, van Kammen M, Tatlisumak T, Krzywicka K, Aguiar de Sousa D, Jood K, Field T, Yaghi S, Coutinho J, Nguyen T. External validation of the SI2NCAL2C score for outcomes following cerebral venous thrombosis. Journal Of Stroke And Cerebrovascular Diseases 2024, 33: 107720. PMID: 38614162, DOI: 10.1016/j.jstrokecerebrovasdis.2024.107720.Peer-Reviewed Original ResearchConceptsCerebral venous thrombosisArea under the receiver operating characteristic curvePoor outcomeVenous thrombosisCentral nervous system infectionInternational retrospective studyNervous system infectionExternal validation cohortFocal neurological deficitsReceiver operating characteristic curvePoor outcome dataLevels of hemoglobinLack of follow-upPoorer outcome scoresConsecutive patientsClinical presentationRetrospective studyValidation cohortIntracerebral hemorrhageNeurological deficitsFollow-upOutcome scoresSystemic infectionLevels of glucoseInternational cohortArtificial Intelligence Predicts Hospitalization for Acute Heart Failure Exacerbation in Patients Undergoing Myocardial Perfusion Imaging
Feher A, Bednarski B, Miller R, Shanbhag A, Lemley M, Miras L, Sinusas A, Miller E, Slomka P. Artificial Intelligence Predicts Hospitalization for Acute Heart Failure Exacerbation in Patients Undergoing Myocardial Perfusion Imaging. Journal Of Nuclear Medicine 2024, 65: jnumed.123.266761. PMID: 38548351, PMCID: PMC11064832, DOI: 10.2967/jnumed.123.266761.Peer-Reviewed Original ResearchConceptsMyocardial perfusion imagingHF hospitalizationHeart failureStress left ventricular ejection fractionPerfusion imagingHF exacerbationPredictive of HF hospitalizationsSPECT/CT myocardial perfusion imagingMyocardial perfusionInternational cohortAcute heart failure exacerbationMedian Follow-UpVentricular ejection fractionReceiver-operating-characteristic curveClinical risk factorsHeart failure exacerbationExternal validation cohortAcute HF exacerbationPrevent HF hospitalizationsImaging parametersCalcium scoreEjection fractionClinical parametersCT scanValidation cohortMulti-center retrospective review of vitiligo-like lesions in breast cancer patients treated with cyclin-dependent kinase 4 and 6 inhibitors
Bang A, Fay C, LeBoeuf N, Etaee F, Leventhal J, Sibaud V, Arbesman J, Wang J, Kwong B. Multi-center retrospective review of vitiligo-like lesions in breast cancer patients treated with cyclin-dependent kinase 4 and 6 inhibitors. Breast Cancer Research And Treatment 2024, 204: 643-647. PMID: 38224427, DOI: 10.1007/s10549-023-07217-2.Peer-Reviewed Original ResearchVitiligo-like lesionsProgression-free survivalSurvival benefitMedian progression-free survivalAssociated with survival benefitSkin-directed therapiesMetastatic breast cancerSun-exposed areasPotential treatment optionCyclin-dependent kinase 4ConclusionClinical characteristicsMild repigmentationRuxolitinib creamTopical ruxolitinibMethodsRetrospective reviewResultsMedian ageClinical characteristicsCase reportTreatment optionsBreast cancerKinase inhibitorsCDK4/6iInternational cohortPatientsQuality of life
2023
Data-Driven Harmonization of 2022 Who and ICC Classifications of Myelodysplastic Syndromes/Neoplasms (MDS): A Study By the International Consortium for MDS (icMDS)
Lanino L, Ball S, Bewersdorf J, Marchetti M, Maggioni G, Travaglino E, Al Ali N, Fenaux P, Platzbecker U, Santini V, Diez-Campelo M, Singh A, Jain A, Aguirre L, Tinsley-Vance S, Schwabkey Z, Chan O, Xie Z, Brunner A, Kuykendall A, Bennett J, Buckstein R, Bejar R, Carraway H, DeZern A, Griffiths E, Halene S, Hasserjian R, Lancet J, List A, Loghavi S, Odenike O, Padron E, Patnaik M, Roboz G, Stahl M, Sekeres M, Steensma D, Savona M, Taylor J, Xu M, Sweet K, Sallman D, Nimer S, Hourigan C, Wei A, Sauta E, D'Amico S, Asti G, Castellani G, Borate U, Sanz G, Efficace F, Gore S, Kim T, Daver N, Garcia-Manero G, Rozman M, Orfao A, Wang S, Foucar M, Germing U, Haferlach T, Scheinberg P, Miyazaki Y, Iastrebner M, Kulasekararaj A, Cluzeau T, Kordasti S, van de Loosdrecht A, Ades L, Zeidan A, Komrokji R, Della Porta M. Data-Driven Harmonization of 2022 Who and ICC Classifications of Myelodysplastic Syndromes/Neoplasms (MDS): A Study By the International Consortium for MDS (icMDS). Blood 2023, 142: 998. DOI: 10.1182/blood-2023-186580.Peer-Reviewed Original ResearchBlast countMost patientsTP53 mutationsTET2 mutationsChromosomal abnormalitiesMore TP53 mutationsBone marrow blastsGene mutationsSF3B1 mutationsClinical decision-making processHigh-risk mutationsMarrow blastsMultilineage dysplasiaPatient characteristicsAML patientsClinical entityInternational cohortSHAP analysisMDS casesPatientsClinical relevanceCytogenetic abnormalitiesClinical settingComplex karyotypeU2AF1 mutations
2022
Natural history of liver disease in a large international cohort of children with Alagille syndrome: Results from the GALA study
Vandriel S, Li L, She H, Wang J, Gilbert M, Jankowska I, Czubkowski P, Gliwicz‐Miedzińska D, Gonzales E, Jacquemin E, Bouligand J, Spinner N, Loomes K, Piccoli D, D'Antiga L, Nicastro E, Sokal É, Demaret T, Ebel N, Feinstein J, Fawaz R, Nastasio S, Lacaille F, Debray D, Arnell H, Fischler B, Siew S, Stormon M, Karpen S, Romero R, Kim K, Baek W, Hardikar W, Shankar S, Roberts A, Evans H, Jensen M, Kavan M, Sundaram S, Chaidez A, Karthikeyan P, Sanchez M, Cavalieri M, Verkade H, Lee W, Squires J, Hajinicolaou C, Lertudomphonwanit C, Fischer R, Larson‐Nath C, Mozer‐Glassberg Y, Arikan C, Lin H, Bernabeu J, Alam S, Kelly D, Carvalho E, Ferreira C, Indolfi G, Quiros‐Tejeira R, Bulut P, Calvo P, Önal Z, Valentino P, Desai D, Eshun J, Rogalidou M, Dezsőfi A, Wiecek S, Nebbia G, Pinto R, Wolters V, Tamara M, Zizzo A, Garcia J, Schwarz K, Beretta M, Sandahl T, Jimenez‐Rivera C, Kerkar N, Brecelj J, Mujawar Q, Rock N, Busoms C, Karnsakul W, Lurz E, Santos‐Silva E, Blondet N, Bujanda L, Shah U, Thompson R, Hansen B, Kamath B, Group T. Natural history of liver disease in a large international cohort of children with Alagille syndrome: Results from the GALA study. Hepatology 2022, 77: 512-529. PMID: 36036223, PMCID: PMC9869940, DOI: 10.1002/hep.32761.Peer-Reviewed Original ResearchConceptsNative liver survivalLarge international cohortAlagille syndromeInternational cohortNLS rateNative liverLiver diseaseMedian total bilirubin levelsEvent-free survival rateNatural historyEarly biochemical predictorsEvident portal hypertensionLiver-related eventsRisks of transplantationMulticenter retrospective studyTotal bilirubin levelsEvaluation of therapyMonths of ageGALA studyHepatic outcomesLiver survivalPortal hypertensionTertiary centerBilirubin levelsRetrospective studyInternational multicenter study comparing demographics, therapy and outcomes in bleeding from Mallory Weiss tears and peptic ulcers
Tham JE, Lynch L, Laursen SB, Laine L, Dalton HR, Ngu J, Redondo-Cerezo E, Schultz M, Murray I, Michell N, Morris AJ, Nielsen MM, Stanley AJ. International multicenter study comparing demographics, therapy and outcomes in bleeding from Mallory Weiss tears and peptic ulcers. Endoscopy International Open 2022, 10: e653-e658. PMID: 35571482, PMCID: PMC9106442, DOI: 10.1055/a-1784-0655.Peer-Reviewed Original ResearchMallory-Weiss tearUpper gastrointestinal bleedingPeptic ulcer bleedingEndoscopic therapyEndoscopic stigmataEpinephrine injectionAcute upper gastrointestinal bleedingInternational multicenter studyPUB patientsGastrointestinal bleedingUlcer bleedingUncommon causeMulticenter studyPeptic ulcerPoor outcomeScope clipInternational cohortClean baseLower riskBaseline parametersPatientsTherapyBleedingCommon modalityMortality
2021
Impact of NAFLD on clinical outcomes in hepatocellular carcinoma treated with sorafenib: An international cohort study.
Howell J, Samani A, Mannan B, Hajiev S, Aval L, Abdelmalak R, Tam V, Bettinger D, Thimme R, Taddei T, Kaplan D, Seidensticker M, Sharma R. Impact of NAFLD on clinical outcomes in hepatocellular carcinoma treated with sorafenib: An international cohort study. Journal Of Clinical Oncology 2021, 39: 289-289. DOI: 10.1200/jco.2021.39.3_suppl.289.Peer-Reviewed Original ResearchImpact of NAFLDOverall survivalHepatocellular carcinomaLiver diseaseNon-alcoholic fatty liver diseaseMultivariable Cox proportional hazardsAdvanced-stage hepatocellular carcinomaLower starting doseSorafenib-related toxicityFatty liver diseaseStage hepatocellular carcinomaCox proportional hazardsLogistic regression modellingStarting doseMedian BMIPrognostic factorsSpecialist centresHCC patientsNAFLDTreatment responseInternational cohortPatientsSorafenibDemographic dataSurvival
2020
Post-Laser Twin Anemia Polycythemia Sequence: Diagnosis, Management, and Outcome in an International Cohort of 164 Cases
Tollenaar LSA, Lopriore E, Faiola S, Lanna M, Stirnemann J, Ville Y, Lewi L, Devlieger R, Weingertner AS, Favre R, Hobson SR, Ryan G, Rodo C, Arévalo S, Klaritsch P, Greimel P, Hecher K, de Sousa MT, Khalil A, Thilaganathan B, Bergh EP, Papanna R, Gardener GJ, Carlin A, Bevilacqua E, Sakalo VA, Kostyukov KV, Bahtiyar MO, Wilpers A, Kilby MD, Tiblad E, Oepkes D, Middeldorp JM, Haak MC, Klumper FJCM, Akkermans J, Slaghekke F. Post-Laser Twin Anemia Polycythemia Sequence: Diagnosis, Management, and Outcome in an International Cohort of 164 Cases. Journal Of Clinical Medicine 2020, 9: 1759. PMID: 32517071, PMCID: PMC7355738, DOI: 10.3390/jcm9061759.Peer-Reviewed Original ResearchTwin anemia-polycythemia sequenceSevere neonatal morbidityPost-laser twin anemia-polycythemia sequenceNeonatal morbidityGestational agePerinatal mortalityPartial exchange transfusionIndependent risk factorMedian gestational agePerinatal mortality rateRisk factor analysisTermination of pregnancyDonor twinExpectant managementInitial surgeryIntrauterine transfusionPerinatal outcomesAntenatal managementSecondary outcomesPrimary outcomeExchange transfusionOptimal treatmentRisk factorsMedian numberInternational cohortDelineating D409H (D448H) homozygous phenotype-genotype in an international cohort of the International Collaborative Gaucher Group Gaucher Registry: Cardiac involvement and early mortality
Eid K, McClain M, Mengel E, Mistry P, Baris-Feldman H. Delineating D409H (D448H) homozygous phenotype-genotype in an international cohort of the International Collaborative Gaucher Group Gaucher Registry: Cardiac involvement and early mortality. Molecular Genetics And Metabolism 2020, 129: s52-s53. DOI: 10.1016/j.ymgme.2019.11.117.Peer-Reviewed Original Research
2019
Ankle-brachial index in patients with intermittent claudication is a poor indicator of patient-centered and clinician-based evaluations of functional status
Johnston AL, Vemulapalli S, Gosch KL, Aronow HD, Abbott JD, Patel MR, Smolderen KG, Shishebor M, Spertus JA, Jones WS. Ankle-brachial index in patients with intermittent claudication is a poor indicator of patient-centered and clinician-based evaluations of functional status. Journal Of Vascular Surgery 2019, 69: 906-912. PMID: 30626552, DOI: 10.1016/j.jvs.2018.07.039.Peer-Reviewed Original ResearchMeSH KeywordsAgedAnkle Brachial IndexAustraliaCost of IllnessFemaleHealth StatusHemodynamicsHumansIntermittent ClaudicationMaleMiddle AgedNetherlandsPatient Reported Outcome MeasuresPeripheral Arterial DiseasePredictive Value of TestsProspective StudiesQuality of LifeRegistriesSeverity of Illness IndexUnited StatesConceptsAnkle-brachial indexPeripheral artery diseasePeripheral Artery QuestionnaireIntermittent claudicationRutherford classificationABI valuesPAQ scoresArtery diseaseImpact of PADHealth statusMean ankle-brachial indexPAD-specific health statusSevere peripheral artery diseasePAQ summary scoreStudy of patientsQuality of lifeHealth status assessmentRutherford classNew onsetPatient's symptomsHemodynamic assessmentLife scoresFunctional statusPatients' perceptionsInternational cohort
2018
The epilepsy bioinformatics study for anti-epileptogenic therapy (EpiBioS4Rx) clinical biomarker: Study design and protocol
Vespa PM, Shrestha V, Abend N, Agoston D, Au A, Bell MJ, Bleck TP, Blanco MB, Claassen J, Diaz-Arrastia R, Duncan D, Ellingson B, Foreman B, Gilmore EJ, Hirsch L, Hunn M, Kamnaksh A, McArthur D, Morokoff A, O'Brien T, O'Phelan K, Robertson CL, Rosenthal E, Staba R, Toga A, Willyerd FA, Zimmermann L, Yam E, Martinez S, Real C, Engel J. The epilepsy bioinformatics study for anti-epileptogenic therapy (EpiBioS4Rx) clinical biomarker: Study design and protocol. Neurobiology Of Disease 2018, 123: 110-114. PMID: 30048805, PMCID: PMC6344322, DOI: 10.1016/j.nbd.2018.07.025.Peer-Reviewed Original ResearchConceptsPost-traumatic epilepsyTraumatic brain injuryIncidence of PTELongitudinal prospective observational studyAnti-epileptogenic therapiesEpilepsy Bioinformatics StudyProspective observational studyFuture interventional studiesModerate-severe TBI patientsHemorrhagic contusionAcute evaluationTBI patientsBlood biomarkersDiscovery of biomarkersInterventional studyBrain injuryElectroencephalographic monitoringObservational studyInternational cohortAnimal modelsClinical biomarkersStudy designBiomarkersMultidisciplinary approachNational InstituteAllogeneic Hematopoietic Stem Cell Transplantation Following the Use of Hypomethylating Agents among Patients with Relapsed or Refractory AML: Findings from an International Retrospective Study
Stahl M, DeVeaux M, Montesinos P, Itzykson R, Ritchie EK, Sekeres MA, Majhail N, Barnard J, Podoltsev NA, Brunner AM, Komrokji RS, Bhatt VR, Al-Kali A, Cluzeau T, Santini V, Roboz GJ, Fenaux P, Litzow M, Fathi AT, Perreault S, Kim TK, Prebet T, Vey N, Verma V, Kobbe G, Bergua J, Serrano J, Gore SD, Zeidan AM. Allogeneic Hematopoietic Stem Cell Transplantation Following the Use of Hypomethylating Agents among Patients with Relapsed or Refractory AML: Findings from an International Retrospective Study. Transplantation And Cellular Therapy 2018, 24: 1754-1758. PMID: 29649620, DOI: 10.1016/j.bbmt.2018.03.025.Peer-Reviewed Original ResearchConceptsHematopoietic stem cell transplantationAllogeneic hematopoietic stem cell transplantationHMA therapyStem cell transplantationComplete remissionCell transplantationUnrelated hematopoietic stem cell transplantationInternational retrospective studyLimited treatment optionsAcute myeloid leukemiaChronic GVHDMedian OSPrimary refractoryRR-AMLConditioning regimenRefractory AMLPatients patientsUnrelated donorsEntire cohortPoor prognosisRetrospective studyTreatment optionsMyeloid leukemiaInternational cohortHypomethylating agent
2017
Whole Exome Sequencing of Patients with Steroid-Resistant Nephrotic Syndrome
Warejko JK, Tan W, Daga A, Schapiro D, Lawson JA, Shril S, Lovric S, Ashraf S, Rao J, Hermle T, Jobst-Schwan T, Widmeier E, Majmundar AJ, Schneider R, Gee HY, Schmidt JM, Vivante A, van der Ven AT, Ityel H, Chen J, Sadowski CE, Kohl S, Pabst WL, Nakayama M, Somers MJG, Rodig NM, Daouk G, Baum M, Stein DR, Ferguson MA, Traum AZ, Soliman NA, Kari JA, El Desoky S, Fathy H, Zenker M, Bakkaloglu SA, Müller D, Noyan A, Ozaltin F, Cadnapaphornchai MA, Hashmi S, Hopcian J, Kopp JB, Benador N, Bockenhauer D, Bogdanovic R, Stajić N, Chernin G, Ettenger R, Fehrenbach H, Kemper M, Munarriz RL, Podracka L, Büscher R, Serdaroglu E, Tasic V, Mane S, Lifton RP, Braun DA, Hildebrandt F. Whole Exome Sequencing of Patients with Steroid-Resistant Nephrotic Syndrome. Clinical Journal Of The American Society Of Nephrology 2017, 13: 53-62. PMID: 29127259, PMCID: PMC5753307, DOI: 10.2215/cjn.04120417.Peer-Reviewed Original ResearchMeSH KeywordsAdolescentAdultAge of OnsetChildChild, PreschoolDNA Mutational AnalysisExome SequencingFemaleGenetic Association StudiesGenetic MarkersGenetic Predisposition to DiseaseHeredityHumansInfantMaleMutationMutation RateNephrotic SyndromePedigreePhenotypePredictive Value of TestsPrognosisYoung AdultConceptsSteroid-resistant nephrotic syndromeNephrotic syndromeWhole-exome sequencingExome sequencingMonogenic causesResistant nephrotic syndromeManagement of treatmentYears of ageCongenital nephrotic syndromeAge of onsetCausative mutationsSyndrome geneKidney transplantationInternational cohortMonogenic genesMolecular genetic diagnosisSyndromePanel sequencingAbstractTextIdentification of mutationsNonconsanguineous familiesPatientsConsanguineous familyGenetic diagnosisDiagnosis
2016
ASSOCIATION BETWEEN PATIENT CHARACTERISTICS AND BASELINE HEALTH STATUS AMONG AN INTERNATIONAL COHORT OF CLAUDICANTS: AN ANALYSIS FROM THE PATIENT-CENTERED OUTCOMES RELATED TO TREATMENT PRACTICES IN PERIPHERAL ARTERIAL DISEASE: INVESTIGATING TRAJECTORIES (PORTRAIT) REGISTRY
Noyes A, Abbott J, Gosch K, Smolderen K, Spertus J, Hyder O, Soukas P, Shishehbor M, Aronow H. ASSOCIATION BETWEEN PATIENT CHARACTERISTICS AND BASELINE HEALTH STATUS AMONG AN INTERNATIONAL COHORT OF CLAUDICANTS: AN ANALYSIS FROM THE PATIENT-CENTERED OUTCOMES RELATED TO TREATMENT PRACTICES IN PERIPHERAL ARTERIAL DISEASE: INVESTIGATING TRAJECTORIES (PORTRAIT) REGISTRY. Journal Of The American College Of Cardiology 2016, 67: 2263. DOI: 10.1016/s0735-1097(16)32264-1.Peer-Reviewed Original Research
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